Preliminary results from phase I/II, first-in-human, open-label study of DNL126 in children with mucopolysaccharidosis type IIIA (MPS IIIA)

Molecular Genetics and Metabolism 2026

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Authors: Elizabeth Jalazo, John A. Bernat, Jimmy Lloyd Holder, Ana-Claire Meyer, Audrey Gill, Nikhil J. Pandya, Jana Matuskova, Carole Ho, Matt D. Troyer, Young S. Oh, Paul Harmatz
DOI: 10.1016/j.ymgme.2025.109481
Cited by: 0

Fields of Study & Subjects

Lysosomal Storage Disorders Research Glycogen Storage Diseases and Myoclonus Virus-based gene therapy research

Publication details

Journal / source
Molecular Genetics and Metabolism
Volume
147
Issue
2
Pages
109481–109481
Publication date
1 February 2026
Language
EN